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Meaningful Patient-Focused Drug Development for Rare Disease and Personalized Medicine
Session Chair(s)
Michelle Campbell, PHD
Associate Director, Stakeholder Engagement and Clinical Outcomes, ON, OND, CDER, FDA, United States
This session will provide a platform to discuss measurement challenges and practical considerations for selection of fit-for-purpose clinical outcome assessments (COAs) to support endpoints in rare disease drug development programs.
Learning Objective : 1. Review commonly used metrics to assess treatment benefit, especially considering survival endpoints. 2. Increase awareness and understanding of a novel statistical approach that has shed new light in the assessment of treatment benefit. 3. Describe ongoing efforts to enhance this methodology and make it available to end-users, such as clinical trialists, physicians, and patients.
Speaker(s)
Generalized Pairwise Comparisons for Benefit/Risk Assessment in Personalized Medicine
Chief Scientific Officer, IDDI, United States
Endpoint Selection and Use of Clinical Outcome Assessments (COAs) in Rare Disease Drug Development: A Regulatory Perspective
Mathematical Statistician, Office of Biostatistics, OTS, CDER, FDA, United States
Selection of Fit-for-Purpose COAs to Support Endpoints in Rare Disease
Vice President, Patient-Centered Science, IQVIA, United States
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